Focused Therapeutic Areas

We are very attentive to the needs of our partners, and recognize that certain therapeutic areas involve additional complexity or high competition, requiring the support of more specialized expertise for a project to progress swiftly and successfully into the clinic.
To this end, we have established focused therapeutic areas, based on our team’s unique expertise, which can provide additional support for projects within their purview. While our drug discovery expertise spans many additional disease areas, these three units ensure that relevant projects benefit from additional know-how and specialized toolkits.

Neuroscience

The Neuroscience unit focuses primarily on neurodegenerative diseases, including Huntington’s Disease, Parkinson’s Disease, Alzheimer’s Disease and ALS. Leveraging both a strong network of academic, industrial and non-profit collaborators as well as extensive expertise in diseases caused by protein misfolding, the Unit supports projects through mechanistic studies and translational research capabilities, and in-house assays developed specifically for the needs of our partners.

These include ultrasensitive biomarker assays for proteins of interest (such as mutant huntingtin, or α-synuclein) in relevant biological samples of both preclinical and clinical samples (e.g. CSF and blood-derived samples). The Unit is seamlessly integrated within IRBM’s drug discovery platform, and can leverage a variety of relevant cell-based models including iPSCs and primary neuronal cultures.

Unit Capabilities

Expertise in neurodegeneration and protein misfolding diseases;

Modulation of toxic protein aggregate conformations;

Modulation of patho-pathways (autophagy, Wnt);

Stratification and efficacy biomarkers.

Oncology

The Oncology Group focuses on fundamental problems in biology of relevance to cancer. The goal of the group is to translate laboratory/project findings into the development of new therapeutic strategies.
Our expertise in cancer and developmental biology, tumor metastasis, signal transduction and patient-derived cancer stem cells support the in vitro and the in vivo oncology platforms suitable for hit validation and MoA studies.

Our internal and external drug discovery projects have the opportunity to benefit from a molecularly annotated tumor cancer stem cell biobank from lung and colorectal patients. The platform, available in-house, is the result of a commercial partnership with the Italian Institute of Health.

Unit Capabilities

Extensive experience in cancer biology, translational approaches

In vitro, in vivo and ex vivo capabilities

Validated, molecularly characterized cancer stem cell models

Strong background in developmental pathways

Experience in xenograft/PDX models

Infectious and Neglected Diseases

The Infectious Diseases Therapeutic Unit focuses on parasitic and viral diseases that affect millions of people in the developing world. Several such illnesses are classified as Neglected Diseases since they affect the world’s poorest people that cannot afford expensive cures. We concentrate our efforts on important diseases such as malaria, sleeping sickness or human African trypanosomiasis (HAT), Chagas disease, schistosomiasis and Zika virus.

Our expertise in the molecular biology of infectious diseases, together with internal capabilities of drug discovery and assay screening aim to develop novel therapeutic agents to face the increasing incidence of diseases which may already be considered global threats.